WO2000078939A3 - Vectors for the treatment of tumors by gene therapy - Google Patents

Vectors for the treatment of tumors by gene therapy Download PDF

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Publication number
WO2000078939A3
WO2000078939A3 PCT/DE2000/002041 DE0002041W WO0078939A3 WO 2000078939 A3 WO2000078939 A3 WO 2000078939A3 DE 0002041 W DE0002041 W DE 0002041W WO 0078939 A3 WO0078939 A3 WO 0078939A3
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WO
WIPO (PCT)
Prior art keywords
vectors
tissue
promoter
specific
prodrug
Prior art date
Application number
PCT/DE2000/002041
Other languages
German (de)
French (fr)
Other versions
WO2000078939A2 (en
Inventor
Dirk Schadendorf
Annette Paschen
Original Assignee
Deutsches Krebsforsch
Dirk Schadendorf
Annette Paschen
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Deutsches Krebsforsch, Dirk Schadendorf, Annette Paschen filed Critical Deutsches Krebsforsch
Publication of WO2000078939A2 publication Critical patent/WO2000078939A2/en
Publication of WO2000078939A3 publication Critical patent/WO2000078939A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention relates to expression vectors for the tissue-specific expression of a gene that codes for a cytotoxic or 'prodrug'-activating protein. Said expression vector comprises the following DNA sequences in a functional combination: a) one or more tissue-specific enhancer elements, for example murine or human tyrosinase enhancer; b) a tissue-specific promoter, for example the tyrosinase promoter or the 'melanoma inhibitory activity' (MIA) promoter; and c) a DNA sequence coding for a cytotoxic or 'prodrug'-activating protein, for example the A chain of the diphtheria toxin or herpes simplex virus thymidine kinase (HSV-TK). These vectors are especially suitable for treating tumors, for example malignant melanoma.
PCT/DE2000/002041 1999-06-21 2000-06-21 Vectors for the treatment of tumors by gene therapy WO2000078939A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE19928342A DE19928342A1 (en) 1999-06-21 1999-06-21 New vector for tissue-specific expression, useful for treating tumors, contains gene for e.g. a cytotoxin under control of tissue-specific promoter and enhancer
DE19928342.7 1999-06-21

Publications (2)

Publication Number Publication Date
WO2000078939A2 WO2000078939A2 (en) 2000-12-28
WO2000078939A3 true WO2000078939A3 (en) 2001-07-26

Family

ID=7911995

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/DE2000/002041 WO2000078939A2 (en) 1999-06-21 2000-06-21 Vectors for the treatment of tumors by gene therapy

Country Status (2)

Country Link
DE (1) DE19928342A1 (en)
WO (1) WO2000078939A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11400039B2 (en) * 2020-01-16 2022-08-02 James W. Hill Changing eye color by gene transduction

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999006576A1 (en) * 1997-08-04 1999-02-11 Calydon, Inc. A human glandular kallikrein enhancer, vectors comprising the enhancer and methods of use thereof
US5871726A (en) * 1995-06-27 1999-02-16 Calydon, Inc. Tissue specific and tumor growth supperssion by adenovirus comprising prostate specific antigen
WO1999018195A2 (en) * 1997-10-03 1999-04-15 Yissum Research Development Company Of The Hebrew University Of Jerusalem Methods and compositions for inducing tumor-specific cytotoxicity

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE165516T1 (en) * 1989-03-21 1998-05-15 Vical Inc EXPRESSION OF EXOGENEOUS POLYNUCLEOTIDE SEQUENCES IN VERTEBRATES
EP0637966B1 (en) * 1992-05-01 1997-08-27 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by the SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Bystander effect tumoricidal therapy
GB9415167D0 (en) * 1994-07-27 1994-09-14 Springer Caroline J Improvements relating to cancer therapy
FR2748032B1 (en) * 1996-04-25 1998-05-22 Rhone Poulenc Rorer Sa TYROSINE HYDROXYLASE GENE DERIVATIVE EXPRESSION SYSTEM

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5871726A (en) * 1995-06-27 1999-02-16 Calydon, Inc. Tissue specific and tumor growth supperssion by adenovirus comprising prostate specific antigen
WO1999006576A1 (en) * 1997-08-04 1999-02-11 Calydon, Inc. A human glandular kallikrein enhancer, vectors comprising the enhancer and methods of use thereof
WO1999018195A2 (en) * 1997-10-03 1999-04-15 Yissum Research Development Company Of The Hebrew University Of Jerusalem Methods and compositions for inducing tumor-specific cytotoxicity

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
MAXWELL I H ET AL: "EXPRESSION OF THE DIPHTHERIA TOXIN A-CHAIN CODING SEQUENCE UNDER THE CONTROL OF PROMOTERS AND ENHANCERS FROM IMMUNOGLOBULIN GENES AS A MEANS OF DIRECTING TOXICITY TO B-LYMPHOID CELLS", CANCER RESEARCH,AMERICAN ASSOCIATION FOR CANCER RESEARCH, BALTIMORE, MD,US, vol. 51, 15 August 1991 (1991-08-15), pages 4299 - 4304, XP000569794, ISSN: 0008-5472 *
PARK BERNARD J ET AL: "Augmentation of melanoma-specific gene expression using a tandem melanocyte-specific enhancer results in increased cytotoxicity of the purine nucleoside phosphorylase gene in melanoma.", HUMAN GENE THERAPY, vol. 10, no. 6, 10 April 1999 (1999-04-10), pages 889 - 898, XP000940967, ISSN: 1043-0342 *
ROBERTSON, MW ET AL: "Use of a tissue-specific promoter for targeted expression of the herpes simplex virus thymidine kinase gene in cervical carcinoma cells", CANCER GENE THER, vol. 5, no. 5, 1998, pages 331 - 336, XP000940909 *
YEW, NS ET AL: "Optimization of plasmid vectors for high-level expression in lung epithelial cells", HUM GENE THER, vol. 8, no. 5, 20 March 1997 (1997-03-20), pages 575 - 584, XP000940966 *

Also Published As

Publication number Publication date
DE19928342A1 (en) 2000-12-28
WO2000078939A2 (en) 2000-12-28

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